title: Development and validation of 11 symptom indexes to evaluate response to chemotherapy for advanced cancer.
abstract: Recent guidance from the FDA discusses patient-reported outcomes as end points in clinical trials. Using methods consistent with this guidance, the authors developed symptom indexes for patients with advanced cancer. Input on the most important symptoms was obtained from 533 patients recruited from NCCN Member Institutions and 4 nonprofit social service organizations. Diagnoses included bladder, brain, breast, colorectal, head and neck, hepatobiliary/pancreatic, kidney, lung, ovarian, and prostate cancers and lymphoma. Physician experts in each of these diseases were also surveyed to differentiate symptoms that were predominantly disease-based from those that were predominantly treatment-induced. Results are evaluated alongside previously published indexes for 9 of these 11 advanced cancers that were created based on expert provider surveys, also implemented at NCCN Member Institutions. Final results are 11 symptom indexes that reflect the highest priorities of people affected by these 11 advanced cancers and the experienced perspective of the people who provide their medical treatment. Beyond the clinical value of such indexes, they may also contribute significantly to satisfying regulatory requirements for a standardized tool to evaluate drug efficacy with respect to symptomatology.
authors.affiliation: Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA. d-cella@northwestern.edu
authors.forename: David
authors.initials: D
authors.lastname: Cella
authors.forename: Sarah K
authors.initials: SK
authors.lastname: Rosenbloom
authors.forename: Jennifer L
authors.initials: JL
authors.lastname: Beaumont
authors.forename: Susan E
authors.initials: SE
authors.lastname: Yount
authors.forename: Diane
authors.initials: D
authors.lastname: Paul
authors.forename: Debra
authors.initials: D
authors.lastname: Hampton
authors.forename: Amy P
authors.initials: AP
authors.lastname: Abernethy
authors.forename: Paul B
authors.initials: PB
authors.lastname: Jacobsen
authors.forename: Karen
authors.initials: K
authors.lastname: Syrjala
authors.forename: Jamie H
authors.initials: JH
authors.lastname: Von Roenn
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D024221:Antineoplastic Protocols; D006801:Humans; D009369:Neoplasms / Q000175:diagnosis* / Q000188:drug therapy*; D018802:Patient-Centered Care; D011788:Quality of Life; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents